Aegerion initiates Phase III trial of Juxtapid/Lojuxta for HoFH-Aegerion Pharma

Aegerion Pharmaceuticals has announced that patient enrollment has been initiated in Japan in a clinical trial of Juxtapid / Lojuxta (lomitapide) as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C) in Japanese patients with Homozygous Familial Hypercholesterolemia (HoFH). The Phase III, open-label trial in Japan is similar in design to the completed Phase III trial conducted in the United States and other countries. Estimated enrollment is between five and 10 adult HoFH patients who are receiving concomitant lipid-lowering therapies, including, in some cases, apheresis.

After a six week run-in period, patients will receive lomitapide for 26 weeks, starting at 5 mg/day and escalating to a maximum dose of 60 mg based on tolerability. After 26 weeks, patients will enter a 30 week safety phase. The primary endpoint is % change in LDL-C levels from baseline to week 26. Secondary endpoints include % change in lipid parameters, long-term safety, and changes in hepatic fat from baseline to week 56. The Japanese regulatory authorities (PMDA) have indicated they will allow the new drug application to be filed following completion of the 26 week efficacy phase.